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First Ever Trial Tests Cholesterol lowering Statins in Children with Rare Genetic Disorder

CardioSmart News

Statins appear safe and effective in children with a rare condition causing extremely high cholesterol, based on results of clinical trial published in the Journal of the American College of Cardiology.

Known as the HYDRA study (A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia), this global trial included children with a rare type of familial hypercholesterolemia.

Familial hypercholesterolemia, often referred to as FH, is a genetic condition that causes abnormally high cholesterol levels beginning at birth. The first form of FH, which is most common, is passed down by one parent and affects 1 in 250 people worldwide. The second type of FH, however, called homozygous familial hypercholesterolemia, occurs when the genetic mutation is passed down by both parents and is much more severe. This rare condition affects 1 in 300,000 to 1 million people and drastically increases risk for heart attack at an early age.

For this reason, experts encourage use of cholesterol-lowering statins as early as possible in children with FH. However, few studies have tested their safety and efficacy in children with homozygous FH.

To learn more, researchers conducted a 6-month clinical trial with 13 children living with this rare genetic condition. Participants were 11 years old on average and came from seven countries in Asia, Europe, the Middle East and North America.

For the first six weeks, participants were randomly assigned to 20 mg of rosuvastatin or an inactive placebo daily, after which they were switched to the opposite group for another six weeks. Participants then knowingly took rosuvastatin for the last 3 months of the study.

After monitoring low-density lipoprotein cholesterol (the “bad” type of cholesterol), researchers found that statins lowered average cholesterol by 85 mg/dl compared to placebo. Participants also experienced few adverse events, none of which were considered serious.

However, the effects of statins varied depending on the type of genetic mutation a child had. For example, statins actually increased cholesterol levels in three children, while one participant’s cholesterol dropped by 55% with statin use. These children had different genetic causes of FH, which appears to have a strong impact on treatment effects.

In this first-ever statin trial in children with homozygous FH, experts are encouraged by findings. The biggest lesson, according to authors, is that statins appear safe and effective in children with homozygous FH. However, findings also show that the impact of statins depends on the genetic cause of FH.

As a result, experts encourage genetic testing in patients with homozygous FH, especially when considering statin use at an early age. It’s likely that children with different genetic causes of FH respond differently to treatments, and it’s important to identify the best possible treatment for each individual patient.

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